Trial Outcomes & Findings for Growth Hormone Use in Adults With Prader-Willi Syndrome (NCT NCT00444964)

NCT ID: NCT00444964

Last Updated: 2021-01-08

Results Overview

Anthropometric Measures and Body Composition

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

11 participants

Primary outcome timeframe

12 and 24 months

Results posted on

2021-01-08

Participant Flow

Participant milestones

Participant milestones
Measure	Primary Cohort Nutropin AQ: 0.0125 mg/kg/day
Overall Study STARTED	11
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Growth Hormone Use in Adults With Prader-Willi Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Primary Cohort n=11 Participants Nutropin AQ: 0.0125 mg/kg/day
Age, Continuous	32.3 years STANDARD_DEVIATION 11.1 • n=39 Participants
Sex: Female, Male Female	6 Participants n=39 Participants
Sex: Female, Male Male	5 Participants n=39 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants
Race (NIH/OMB) Asian	0 Participants n=39 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants
Race (NIH/OMB) Black or African American	0 Participants n=39 Participants
Race (NIH/OMB) White	11 Participants n=39 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants

PRIMARY outcome

Timeframe: 12 and 24 months

Population: All patients enrolled

Anthropometric Measures and Body Composition

Outcome measures

Outcome measures
Measure	Primary Cohort n=11 Participants Nutropin AQ: 0.0125 mg/kg/day
Body Composition Baseline	51.2 percentage of body fat Standard Deviation 2.1
Body Composition 12 Months	48.5 percentage of body fat Standard Deviation 2.2
Body Composition 24 Months	52.1 percentage of body fat Standard Deviation 2.3

PRIMARY outcome

Timeframe: 12 and 24 months

Population: All patients enrolled

Insulin-like growth factor I- IGF-1

Outcome measures

Outcome measures
Measure	Primary Cohort n=11 Participants Nutropin AQ: 0.0125 mg/kg/day
Blood Chemistry Baseline	93 ng/ml Standard Deviation 37.6
Blood Chemistry 12 months	385 ng/ml Standard Deviation 48.6
Blood Chemistry 24 Months	140 ng/ml Standard Deviation 53.1

PRIMARY outcome

Timeframe: 12 and 24 months

Population: All patients enrolled

Duration of daily physical activity

Outcome measures

Outcome measures
Measure	Primary Cohort n=11 Participants Nutropin AQ: 0.0125 mg/kg/day
Physical Activity Baseline	177 Minutes per Day Standard Deviation 27
Physical Activity 12 Months	256 Minutes per Day Standard Deviation 33
Physical Activity 24 Months	215 Minutes per Day Standard Deviation 49

Adverse Events

Primary Cohort

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Primary Cohort n=11 participants at risk Nutropin AQ: 0.0125 mg/kg/day
Musculoskeletal and connective tissue disorders Fall	9.1% 1/11 • Number of events 1 • 24 months

Other adverse events

Adverse event data not reported

Additional Information

Jaylene Weigel

Children's Mercy Hospital

Phone: 816-701-1359

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place